TRIAL SYNOPSIS

STUDY TYPE:

TITLE OF STUDY:

STUDY CENTERS:

Single site; LA Biomed, Harbor-UCLA Medical Center

NUMBER OF SUBJECTS:

20 Patients

STUDY DURATION:

Study will be concluded 12 months after entry of each patient into the study.

STUDY OBJECTIVE:

To evaluate the safety and efficacy of autologous bone marrow mononuclear cells concentrated using an FDA-approved closed-system device, the Arteriocyte Magellan® device, and administered via intracavernosal injection to 20 patients < 80 years of age who have been diagnosed with vasculogenic erectile dysfunction. Safety will be determined based on reporting of adverse events and blood chemistry panels evaluated at baseline and at follow-up visits at one, three, and six months post-treatment. Efficacy will be evaluated by baseline vs. follow-up comparisons conducted at one, three, and six months post-treatment, evaluating parameters of erectile function; namely, the International Index of Erectile Function (IIEF) questionnaire, Doppler ultrasonography, and dynamic infusion cavernosometry. Additionally, at twelve months post-treatment, patients will be interviewed for adverse events self-reporting and completion of the IIEF questionnaire. The study will determine whether intracavernosal injection of bone marrow mononuclear cells, a rich source of stem cells with cellular repair and regenerative functions, is a safe and clinically feasible approach for improving erectile function.

STUDY RATIONALE:

Bone marrow is enriched for cells with regenerative potential, including mesenchymal stem cells (MSC), which accelerate healing of damaged tissue. The possibility of using bone marrow mononuclear cells in the treatment of ED is enticing since stem/progenitor cell populations are known to secrete various growth factors, possess anti-inflammatory activities, and can differentiate into cells that reconstruct the penile architecture. The therapeutic activities of bone marrow-derived cells as well as stem cells from other sources have been established in animal models of erectile dysfunction [1-8]. This study will examine the safety and efficacy intra-cavernosal injection of bone marrow mononuclear cells in a clinical setting.

Erectile dysfunction (ED) in the aging man) is primarily characterized by abnormalities of the penile vascular system, There is either a problem with corporal veno-occlusive dysfunction (venous leakage resulting from loss of integrity of the corporal smooth muscle cells [9, 10] or defective arterial inflow via the cavernosal arteries. In the penile vascular system, the corporal smooth muscle is responsible for trapping the blood delivered to the corpora cavernosa via the arterial system. Loss of smooth muscle mass is believed to be the major culprit in the inability to maintain erections due to impaired storage of blood in the corporal sinusoids once inflow of blood into the penis begins [10]. Moreover, as men age, the development of atherosclerosis combined with or without the aging related arterial vascular smooth muscle cell loss and and resultant fibrosis can translate into impaired arterial inflow into the penis [11]. For this study, vasculogenic dysfunction refers to someone who has either arteriogenic and/or venogenic evidence as a cause of their ED.

For therapeutic purposes, bone marrow mononuclear cells can be extracted and concentrated in closed system devices that are already being used under the practice of medicine for a variety of indications. In this study, we are seeking to explore the safety and feasibility of bone marrow cells harvested using Arteriocyte’s Magellan® device and administered by intracavernosal injection to patients with ED who are over 18 years of age and have a vasculogenic (arterial and/or venogenic ) cause of their ED.

STUDY ENDPOINTS:

Primary Outcome Measures:

Safety and tolerability based on number and severity of adverse events [Time frame: months 1, 3, 6 and 12]